Spark Resumes Enrollment Into SPK-3006 Clinical Studies for LOPD, Stopped for COVID-19

Spark Resumes Enrollment Into SPK-3006 Clinical Studies for LOPD, Stopped for COVID-19
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Spark Therapeutics has resumed enrollment into two international clinical studies testing SPK-3006, the company’s investigational gene therapy for Pompe disease.

Recruitment of adults with late-onset Pompe disease (LOPD) into both studies — the seroprevalence SPK-GAA-100 study (NCT03893240) and the Phase 1/2 RESOLUTE trial (NCT04093349) — was voluntarily suspended by Spark in March for safety reasons due to the COVID-19 pandemic. That decision was made “in an abundance of caution” following consultations with investigators, advocacy leaders, and healthcare professionals, the company said.

“This voluntary suspension was solely related to the COVID-19 (SARS CoV-2) pandemic and by suspending enrollment we aimed to minimize the risk of exposure to COVID-19 for participants who would have been traveling to and from investigational sites,” the company said in a press release.

Now, after discussing with Pompe experts, consulting the FDA’s guidelines of safe conduct of clinical trials, and re-assessing safety measures currently in place to minimize and halt the spread of COVID-19, Spark (part of Roche) decided to reopen enrollment into both studies of SPK-3006.

The investigational gene therapy is being developed for people with late-onset Pompe, whose symptoms — most notably muscle weakness — began later in life, during childhood or adulthood.

The seroprevalence SPK-GAA-100 study will investigate whether LOPD patients currently on enzyme replacement therapy (ERT) have neutralizing antibodies that may interfere with SPK-3006’s effectiveness. The scientists also intend to cull information on Pompe disease features, including liver and muscle health, to help design a future gene therapy trial.

This trial now will continue to enroll participants at 18 sites across the U.S. and Europe. More information can be found here.

Meanwhile, RESOLUTE — also known as SPK-GAA-101 — is recruiting 20 participants on ERT in the same locations as the SPK-GAA-100 study. This Phase 1/2 trial aims to evaluate the safety, tolerability, and efficacy of a single infusion of SPK-3006 in adults with LOPD. It is expected to conclude in August 2023.

SPK-3006 is an experimental gene therapy that uses a harmless adeno-associated virus (AAV) to deliver a genetically modified version of the GAA gene, which is faulty in people with Pompe, directly into the liver cells. The therapy is given in a single intravenous (into-the-vein) infusion.

Through this mechanism, SPK-3006 is expected to increase the blood levels of the enzyme acid alpha-glucosidase (GAA), which is either absent or not working in these patients. Increasing the GAA levels help ease Pompe symptoms.

Earlier data from preclinical studies showed that when given to a mouse model of Pompe, SPK-3006 increased the animals’ lifespan, reduced the buildup of glycogen (the sugar molecule that accumulates in the absence of GAA), and improved heart, respiratory, and muscle function.

In non-human primate models, a single administration of three increasing doses of SPK-3006 led to a dose-dependent increase in GAA levels in the blood, with no treatment-related toxicities through at least six months.

Spark said it is continuing to evaluate all aspects of the two studies that may impacted by COVID-19. Steps have been taken to minimize visits to study sites, and site personnel will conduct all COVID-19-related screening procedures mandated by the government and the health care systems.  Additional safety measures may be initiated, the company said.

 

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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José holds a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.

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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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